Patient-Reported Outcomes (PROs) play an important role in patient-centered healthcare and in clinical research. Here we will go in depth into what PROs are, how they are used in measuring treatment impact and the importance of PROs in drug development.
Definition and Objectives of PROs
Patient-reported outcomes (PROs) are information about a patient’s health that comes directly from the patient and can include almost any health-relevant facts or descriptions. Often, PROs are used to gather information on topics that are difficult or unduly invasive to measure, such as descriptions of symptoms, mental or physical side effects of treatment, degree of satisfaction with care, severity of pain or limitations in physical functioning.
The Role of PROs in Modern Healthcare
PROs are a direct way of listening to patient experiences, measuring treatment impact and helping improve patient quality of life. They are a key part of the realignment of health professions toward patient-centered healthcare. Additionally, PROs in clinical research and PROs in drug development are a means of considering patient perspectives in experimental care settings, and they help give the full picture of how novel therapies impact patients.
The Importance of PROs in Measuring Treatment Impact
The medical profession is undergoing a reorientation toward patient-centered healthcare. A shift of this kind naturally places emphasis on patient experience, and data from PROs is key to fully understanding how treatment affects a patient.
Enhancing Patient-Centered Care
Patient-centered healthcare is care delivery that makes sure that patients are involved in every decision about their care. This kind of care also goes beyond the clinical aspects and extends to the mental, emotional, social and spiritual components of the patient experience. PROs are vital because they give clinicians a more complete understanding of how treatment impacts patients, allowing patients and providers to tailor care to the specific case and be partners in making a care plan.
Evaluating Quality of Life and Symptom Burden
Quality of life considerations are a key part of patient-centered healthcare. PROs can be used to monitor the effects and progression of a health concern. Symptoms often appear in clusters or sets, and collecting PROs across a patient population can make it easier to identify groupings of symptoms in clinical trials and ordinary care settings alike. When symptom patterns or associations are identified, that makes it easier for the care team to predict the course of treatment and to plan accordingly.
Informing Clinical Decision-Making
PROs (or frequently, electronic patient-reported outcomes or ePRO) provide real-time data to the care team from the patient, recording positive and negative changes. This information is useful to clinicians because it lets them make decisions with a clearer view of patient physical function, pain, anxiety and other domains. When care decisions are made in view of this information, patients are more likely to follow planned courses of treatment and have better end results.
Common Methodologies for Collecting PROs
When clinicians want to collect PROs from patients, there are a few common tools and techniques for doing so. Together, these are called PRO data collection methods.
Standardized PRO Questionnaires
PRO questionnaires are often consistent (i.e. standardized) data collection instruments used across healthcare systems or practice areas. Sometimes the questionnaires address generally applicable concerns, such as how the patient’s day has been, or their mobility and self-care over the last week or month. PRO questionnaires can also be targeted to specific patient groups or conditions, such as people who have undergone knee replacement surgery or are ill with long covid.
Electronic PRO (ePRO) Tools
Electronic patient-reported outcomes (ePRO) are PROs submitted through digital applications – such as smartphone apps or a patient portal set up by the healthcare provider. ePROs have the advantage of faster processing times and increased accuracy, since the data is transferred directly into computer systems and there is no risk of transcription errors. ePRO tools can also send reminders to submit information and patients can use the tools from anywhere, which yields more complete data when measuring treatment impact.
Patient Diaries and Self-Assessments
Patients may also write up their experiences, often with prompts from the healthcare team. Patients may be asked to record how they are feeling generally, or they may be asked about more specific aspects of their experience. Common starting points for patient diaries include assessments of whether the patient can walk for a certain amount of time or to talk about their physical and emotional conditions in a given recent timeframe.
Data Sources for PROs
So what exact situations are contexts for PROs? Here we’ll take a look at a variety of circumstances where PROs may be collected, including PROs in clinical research or PROs in drug development.
Clinical Trials
The experimental nature of drugs and therapies, plus stringent regulatory requirements, means that researchers conducting clinical trials need to be highly attuned to the experience of trial subjects (that is, patients). PROs in clinical research help provide the level of attention required for the development of new therapies, and oversight bodies are increasingly mandating that trial protocols include PRO data collection methods and PRO endpoints.
Patient Registries
Collecting electronic patient-reported outcomes (ePRO) and adding them to patient registries gives physicians access to patient experience data generated in the course of real clinical practice. Registry data helps make data on treatment more generalizable because patient cases in registries originate outside the carefully controlled settings of clinical trials. As such, ePRO in registries can give insight to physicians facing unique or challenging cases in their practice because they can see information from similar cases and across populations.
Electronic Health Records (EHRs)
Integrating PROs – and especially ePRO – into electronic health records (EHRs) is becoming routine, even outside of the research setting. ePROs are now common parts of patient files and are used to facilitate even routine care. Patients can report their experiences and the care team can draw on that information when considering treatment options, increasing the likelihood of successful treatment outcomes.
Mobile Health Applications
Mobile health applications can be valuable tools for collecting and using ePROs, especially in telehealth settings. Apps can be used to track symptoms and monitor progress, and this data can be straightforwardly transferred to physicians to help guide care plans. Apps may be designed in-house by the healthcare providers, or patients may also use third-party tools to record PROs and share this information with physicians.
Challenges in Implementing PROs
PROs are not perfect. There can be issues because the questionnaires, survey instruments and PRO data collection methods may not address all relevant information, and patients themselves sometimes do not share everything that they could.
Ensuring Accuracy and Reliability of Patient Data
Meausring treatment impact and other PRO use cases often have a subjective element. PRO data collection methods can also have shortcomings (e.g. questionnaires that are unclear or do not address all relevant topics), and patients may also experience participant fatigue or find it tedious to repeatedly complete the same PRO reporting tasks.
Patient Engagement and Compliance
Patients may forget to fill out PRO forms or find it monotonous and stop doing it. Patients might also leave out useful information or, due to disinterest or confusion about the purpose of PROs, just “check the boxes” and give incomplete or inaccurate information.
Standardization Across Different Studies
A variety of PRO data collection methods exists, with variation across companies, hospitals and research groups. This can lead to difficulty in synthesizing data because of the different ways used to measure the same thing. Standardizing PRO data collection methods across studies and practice groups is a challenge when implementing PROs.
Applications of PROs in Healthcare
Let’s have a look at how PROs can be applied in patient-centered healthcare.
Assessing Treatment Efficacy and Safety
PROs complement traditional clinical measures of efficacy and safety. For instance, oncologists will of course be interested in the comparative survival rates of surgical and radiation interventions. Even if these figures are comparable, PROs can provide insight on quality of life matters – such as mobility issues or chronic pain after treatment – that other metrics do not capture.
Monitoring Chronic Diseases
Chronic diseases are especially practicable areas for PRO application because of their long timeframes. PROs give a longitudinal view of symptom evolution and treatment impact in chronic cases, allowing clinicians to identify trends and make treatment more responsive to the particular characteristics of a given patient.
Improving Drug Development and Regulatory Approvals
PROs in drug development are taking on growing importance. Their relevance for evaluating and responding to patient needs in a timely manner during clinical trials has long been recognized. In fact, in the United States the FDA requires PRO endpoints in many clinical trials when seeking approval for new drugs.
Case Studies: Successful Use of PROs
ESR has a record of success in collecting PROs for clinical and research uses. For instance, our firm oversees PRO tracking for multiple myeloma patients in a south European country. In that project, 75 haematologists need to collect data in 10-12 patient report forms per quarter, with quotas by region and type of setting. Through its PRO tools, ESR has kept these respondents engaged and has achieved a 90% wave-to-wave repetition rate among respondents for the 10+ years that this tracking project has been active.
Likewise, ESR is off to a good start in a more recent PRO collection project. In this case, the work area is a metastatic urothelial cancer study being conducted in 5 Latin American countries. There are approximately 50 clinician respondents per country who are to collect 3 to 5 patient report forms with PROs. ESR oversaw the successful completion of the first wave in that project, and the second wave will be completed in spring 2025.
Advantages of Using PROs
Even beyond cases where PROs are necessary as regulator-mandated endpoints, incorporating PROs into healthcare provides benefits to patients, families and clinicians.
Improved Clinical Guidelines and Care Pathways
When patients see that physicians are taking their concerns seriously, they are more likely to be partners in making and following care plans. Additionally, clinicians who consider PROs will have data to help them tailor treatments and practices to the particular patient, increasing the chances of both compliance with the treatment plan and a long-term healthier patient.
Enhanced Shared Decision-Making
PROs – especially ePROs – integrate patient experience information into their file with the provider. This combination of patient experience plus clinical expertise gives both parties key information so they can decide on a course of action as partners in care.
Why choose ESR Research to measure the impact of treatments with PROs
ESR Research offers comprehensive and intuitive solutions for collecting and implementing PRO data. Whether you’re working with PROs in clinical trials, PROs in drug development or in the course of routine care, patients and clinicians alike will find ESR Research’s tools easy to use and comprehensive in their output.
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